Clinical Trial: A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment
Study Status: Completed
Recruit Status: Completed
Study Type: Observational
Official Title: Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment
Brief Summary: The purpose of this study is to investigate the relationship of anti-erythropoietin antibodies to the clinical course and outcome of pure red cell aplasia (PRCA) in participants currently or previously treated with recombinant human erythropoietin.
Detailed Summary: This is a multicenter (study conducted at multiple sites), observational (study in which the investigators/physicians observe the participant's data and measure their outcomes) study. Approximately 150 participants will be enrolled in this study. The study consists of an initial observation phase and extended observation period. An initial observation phase starting at enrollment and ending when 24 months have elapsed since the date of loss of efficacy (LOE), supplemented with retrospective data collection for the period between LOE date and date of enrollment in the study. Participants remaining epoetin alfa (EPO-Ab) positive 24 months after LOE will enter a 2-year extended observation period. Study visits will take place every month during the initial observation phase and data will be collected every 6 months during the extended observation phase. Safety evaluations will include assessment of adverse events, clinical laboratory tests, vital signs, and physical examination which will be monitored throughout the study. The total study duration for each participant will be approximately for 4 years.
Sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Current Primary Outcome:
- Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase) [ Time Frame: Up to 24 months after the date of loss of efficacy ]The PRCA outcome is measured by anti-epoetin alfa qualitative test. Persistence of PRCA is defined as: 1) absolute reticulocyte count less than 30,000 per cubic millimeter; and/or 2) no reversal of erythroblastopenia on repeated bone marrow testing. Resolution of PRCA is defined as: 1) absolute reticulocyte count greater than or equal to 30,000 per cubic millimeter; and/or 2) reversal of erythroblastopenia on repeated bone marrow testing.
- Number of participants with pure red cell aplasia outcome (Extended observation phase) [ Time Frame: Up to 2 years after the enrollment in the extended observation phase ]Participants remaining anti-epoetin alfa positive 24 months after loss of efficacy will enter in the extended observation period.
- Overall clinical outcome of pure red cell aplasia (Initial observation phase) [ Time Frame: Up to 24 months after the date of loss of efficacy ]The overall clinical outcome is evaluated by anti-epoetin alfa qualitative test. Overall clinical status will be recorded at each visit in the initial and extended observation phases using a categorical scale (improved, same, worsened, death). In case of death, the date and cause of death along with the the date and cause of death will be recorded.
- Overall clinical outcome of pure red cell aplasia (Extended observation phase) [ Time Frame: Up to 2 years after the enrollment in the extended observation phase ]
Original Primary Outcome:
Current Secondary Outcome:
- Different treatment modalities with pure red cell aplasia outcome (Initial observation phase) [ Time Frame: Up to 24 months after the date of loss of efficacy ]
- Different treatment modalities with pure red cell aplasia outcome (Extended observation phase) [ Time Frame: Up to 2 years after the enrollment in the extended observation phase ]
- Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome [ Time Frame: Period between LOE date and date of enrollment in the study ]This data will be collected retrospectively and the date of LOE will be determined by the sponsor based upon reported data. PRCA duration groups will be summarized by potential risk factors to evaluate the relationship of risk factors to the duration of PRCA.
Original Secondary Outcome:
Information By: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Dates:
Date Received: September 13, 2005
Date Started: February 2004
Date Completion:
Last Updated: April 29, 2013
Last Verified: April 2013
